MDS and Allogeneic Stem Cell TransplantationNCT04801563
Allogeneic HSCT in MDS Patients Based on Risk According to R-IPSS
A study testing MDS and AlloHSCT in people with MDS and Allogeneic Stem Cell Transplantation, run by University Of Perugia at 1 site in Italy.
Trial summary
An "intention-to-treat" study to evaluate the impact of allogeneic HSCT with Total Marrow and Lymphoid irradiation (TMLI), followed by Treg/Tcon adoptive immunotherapy, on overall survival in patients affected by Myelodysplastic Syndrome (MDS), according to IPSS-R. Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- MDS and Allogeneic Stem Cell Transplantation
- What is being tested?
- MDS and AlloHSCT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteItaly
- Study dates
- 20 Sep 2019to 19 Sep 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients affected by Myelodysplastic Syndrome with IPSS-R INT, HIGH or VERY HIGH;
- Age <71 years;
- Signature of the informed consent.
Who cannot join
- Patients affected by Myelodysplastic Syndrome with IPSS-R VERY LOW or LOW;
- Patients affected by Myelodysplastic Syndrome/Myeloproliferative Neoplasm;
- Age >70 years;
- No signature of the informed consent
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
University of Perugia
Perugia, PG, 06132
Antonio Pierini, MD, +390755784147, antonio.pierini@unipg.it
MERLUZZI MARA, +393482200239, maramerluzzi@libero.it
About this study
Official title: Allogeneic Transplantation in Patients With Myelodysplastic Syndrome Based on Risk According to Revised-International Prognostic Scoring System (R-IPSS)
An "intention-to-treat" study to evaluate the impact of allogeneic HSCT with Total Marrow and Lymphoid irradiation (TMLI), followed by Treg/Tcon adoptive immunotherapy, on overall survival in patients affected by Myelodysplastic Syndrome (MDS), according to IPSS-R.
Read the detailed description
MDS patients with Intermediate, High, or Very-High R-IPSS risk will be enrolled. Allogeneic stem cell transplantation with regulatory and conventional T cell adoptive immunotherapy will be proposed to eligible High and Very-High risk MDS patients. Intermediate risk MDS patients will be assessed for risk modification every six months and moved to transplant in case of increased risk. Intermediate risk MDS patients will be also evaluated for the presence of molecular alterations (TP53, ASXL1, RUNX1). Overall survival of the entire cohort will be assessed as primary endpoint. Incidence of Treatment Related Mortality, acute Graft versus Host Disease, chronic Graft versus Host Disease, Relapse will be also assessed.
- Being tested
- MDS and AlloHSCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MDS and AlloHSCTOther
Allogeneic stem cell transplantation, based on TMLI and followed by infusion of donor regulatory T cells and conventional T cells and purified donor CD34+ hematopoietic stem cells
| Study group | Role | What they receive |
|---|---|---|
| SINGLE ARM | Other | Not described Receives: Other: MDS and AlloHSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival
We will evaluate overall survival in patients with intermediate/High/very-High risk MDS
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2021
- Study start20 Sep 2019
- Primary completion19 Sep 2025 (estimated)
- Study completion19 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for MDS and Allogeneic Stem Cell TransplantationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Of Perugia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Andrea Velardi, University Of Perugia
- Investigators
- Andrea Velardi, MD, Principal investigator, University Of Perugia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04801563
- Sponsor's protocol ID
- 03/19
- Status last verified
- Mar 2021
- First submitted
- 28 Feb 2021
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Di Ianni M, Falzetti F, Carotti A, Terenzi A, Castellino F, Bonifacio E, Del Papa B, Zei T, Ostini RI, Cecchini D, Aloisi T, Perruccio K, Ruggeri L, Balucani C, Pierini A, Sportoletti P, Aristei C, Falini B, Reisner Y, Velardi A, Aversa F, Martelli MF. Tregs prevent GVHD and promote immune reconstitution in HLA-haploidentical transplantation. Blood. 2011 Apr 7;117(14):3921-8. doi: 10.1182/blood-2010-10-311894. Epub 2011 Feb 3.
PubMed 21292771Martelli MF, Di Ianni M, Ruggeri L, Falzetti F, Carotti A, Terenzi A, Pierini A, Massei MS, Amico L, Urbani E, Del Papa B, Zei T, Iacucci Ostini R, Cecchini D, Tognellini R, Reisner Y, Aversa F, Falini B, Velardi A. HLA-haploidentical transplantation with regulatory and conventional T-cell adoptive immunotherapy prevents acute leukemia relapse. Blood. 2014 Jul 24;124(4):638-44. doi: 10.1182/blood-2014-03-564401. Epub 2014 Jun 12.
PubMed 24923299
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Transplantation in Patients With Myelodysplastic Syndrome Based on Risk According to Revised-International Prognostic Scoring System (R-IPSS)
- ClinicalTrials.gov ID
- NCT04801563
- Acronym / sponsor ID
- 03/19
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- University Of PerugiaUniversity, hospital or other
- Study start
- 20 Sep 2019 (actual)
- Primary completion
- 19 Sep 2025 (estimated)
- Study completion
- 19 Sep 2025 (estimated)
- First posted
- 17 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An "intention-to-treat" study to evaluate the impact of allogeneic HSCT with Total Marrow and Lymphoid irradiation (TMLI), followed by Treg/Tcon adoptive immunotherapy, on overall survival in patients affected by Myelodysplastic Syndrome (MDS), according to IPSS-R.
Detailed description
MDS patients with Intermediate, High, or Very-High R-IPSS risk will be enrolled. Allogeneic stem cell transplantation with regulatory and conventional T cell adoptive immunotherapy will be proposed to eligible High and Very-High risk MDS patients. Intermediate risk MDS patients will be assessed for risk modification every six months and moved to transplant in case of increased risk. Intermediate risk MDS patients will be also evaluated for the presence of molecular alterations (TP53, ASXL1, RUNX1). Overall survival of the entire cohort will be assessed as primary endpoint. Incidence of Treatment Related Mortality, acute Graft versus Host Disease, chronic Graft versus Host Disease, Relapse will be also assessed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SINGLE ARM | Other | Other: MDS and AlloHSCT |
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients affected by Myelodysplastic Syndrome with IPSS-R INT, HIGH or VERY HIGH;
- Age <71 years;
- Signature of the informed consent.
Key exclusion criteria (4 listed)
- Patients affected by Myelodysplastic Syndrome with IPSS-R VERY LOW or LOW;
- Patients affected by Myelodysplastic Syndrome/Myeloproliferative Neoplasm;
- Age >70 years;
- No signature of the informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MDS and AlloHSCTAllogeneic stem cell transplantation, based on TMLI and followed by infusion of donor regulatory T cells and conventional T cells and purified donor CD34+ hematopoietic stem cells | Other | SINGLE ARM |
Primary outcome
overall survival
Time frame: 4 years
We will evaluate overall survival in patients with intermediate/High/very-High risk MDS
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- Results publication: PubMed 21292771Di Ianni M, Falzetti F, Carotti A, Terenzi A, Castellino F, Bonifacio E, Del Papa B, Zei T, Ostini RI, Cecchini D, Aloisi T, Perruccio K, Ruggeri L, Balucani C, Pierini A, Sportoletti P, Aristei C, Falini B, Reisner Y, Velardi A, Aversa F, Martelli MF. Tregs prevent GVHD and promote immune reconstitution in HLA-haploidentical transplantation. Blood. 2011 Apr 7;117(14):3921-8. doi: 10.1182/blood-2010-10-311894. Epub 2011 Feb 3.
- Results publication: PubMed 24923299Martelli MF, Di Ianni M, Ruggeri L, Falzetti F, Carotti A, Terenzi A, Pierini A, Massei MS, Amico L, Urbani E, Del Papa B, Zei T, Iacucci Ostini R, Cecchini D, Tognellini R, Reisner Y, Aversa F, Falini B, Velardi A. HLA-haploidentical transplantation with regulatory and conventional T-cell adoptive immunotherapy prevents acute leukemia relapse. Blood. 2014 Jul 24;124(4):638-44. doi: 10.1182/blood-2014-03-564401. Epub 2014 Jun 12.
Related trials for MDS and Allogeneic Stem Cell Transplantation
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Not yet recruitingPhase 1
Maintenance Treatment With Imetelstat in Patients With High-Risk Myeloid Neoplasms After Allo-SCT
High-risk Myeloid Malignancy, Allogenic Stem Cell Transplantation
RecruitingPhase 1
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrea Velardi, MD principal investigator
University Of Perugia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MDS and AlloHSCT in people with MDS and Allogeneic Stem Cell Transplantation, run by University Of Perugia at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Antonio Pierini, MD (+390755784147, antonio.pierini@unipg.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall survival (time frame: 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04801563, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2021.